Biologics

Panel Backs Sparks Gene Therapy for Vision Loss

FDA's Cellular, Tissue and Gene Therapies Advisory Committee has voted unanimously (16 to 0) to recommend approval for a Spark Therapeutics BLA for Luxturna (voretigene neparvovec) for treating patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy. A briefing document released in advance of the meeting noted that the gene therapy met the main criteria for efficacy. In the key study, the reviewers said, patients had a significant two-point or more improvement in light levels in both eyes, but checking visual acuity showed that patients failed to demonstrate a significant improvement in that secondary endpoint.

The therapy has a priority review and a 1/12/2018 user fee review action deadline. It has received orphan drug, breakthrough therapy and rare pediatric disease designations from FDA.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome